Genomic Medicine: Catch The Gene Therapy Wave

Summary

• Kymriah and Yescarta, the first two U.S.-approved autologous T-cell immunotherapies, are commercially available in 2018.
• Three leading gene therapy companies have been acquired in the last nine months for a combined $30 billion.
• Catalysts over the next year are poised to drive group valuations.

Genomic medicine is the future of healthcare. Spawned by the Human Genome Project (“HGP”), this precise and personal approach to medicine is an emerging discipline with enormous disruptive potential. Though nascent, genomic medicine is showing signs of being the transformative force that has been sought by Silicon Valley and DC politicians for many years. This article kicks off a series on cell and gene therapy and genome (aka gene) editing. Companies in this macro segment will be tagged as follows:

• Big Pharma represents the pharmaceutical and large-cap biotechnology companies that are actively engaged in acquiring or collaborating with gene therapy companies. Companies referenced later in this article are Celgene (CELG), Gilead (GILD), Novartis AG (NVS) and Pfizer (PFE).
• Gene and Cell Therapy is the superset representing companies whose primary purpose is pursuing cell therapies, gene therapies, gene regulation or genome editing. The companies tracked include Abeona Therapeutics (ABEO), bluebird bio (BLUE), Audentes Therapeutics (BOLD), AveXis (AVXS), Cellectis S.A. (CLLS), CRISPR Therapeutics (CRSP), Editas Medicine (EDIT), Homology Medicines (FIXX), Intellia Therapeutics (NTLA), Spark Therapeutics (ONCE), uniQure N.V. (QURE), REGENXBIO (RGNX), Sangamo Therapeutics (SGMO) and Voyager Therapeutics (VYGR).
• Genome Editing is a subset of the Gene and Cell Therapy group. The five companies tagged with Genome Editing are Cellectis, CRISPR, Editas, Intellia and Sangamo. Both bluebird bio and Homology Medicines are likely to be included in this group in the next six months. Bluebird acquired privately held Precision Genome Engineering "Pregenen" in 2014, and has been quietly in early research mode. This will be explored further in a future article. Homology is also pursuing genome editing, but further research needs to be done to assess its status.

Healthcare spending

That healthcare spending accounts for 17.9% of the U.S. Gross Domestic Product is enlightening, yet the realization this economic mix was only

Analyst’s Disclosure:I am/we are long ATRA, CELG, GILD, RGNX, SGMO. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.